The goal of gene therapy for glaucoma is to modulate biochemical/physiological processes by up- or down-regulating specific targets. Developing gene delivery systems to deliver nucleic acid drugs to the eye has become a challenge. There are two general methods of delivery, including non-viral vectors and viral vectors. Both approaches have advantages and disadvantages, and the choice of a safe and efficient delivery system is of great importance for gene therapy.
A successful gene therapy strategy requires a safe and effective gene delivery system. Ace Therapeutics offers gene delivery system development services for glaucoma gene therapy, including two major options, viral and non-viral vectors, to ensure the transfer of genes to target cells and to advance the translational application of gene therapy.
Gene Delivery Strategy in Glaucoma
Fig. 1 Different ocular target tissues for glaucoma gene therapy. (Kaufman, et al. 2018)
The eye is a small and complex organ with multiple layers of biological barriers. In glaucoma treatment, it is necessary to deliver drugs around the barrier to the targeted tissues to achieve a therapeutic effect. The same is true for gene therapy, where we can use different delivery systems to achieve the effect of targeted delivery.
There are two general approaches to gene delivery, including viral and non-viral vectors. Viral vectors are efficient vectors but have safety issues. Common viral vectors include adenoviruses, adeno-associated viruses, retroviral vectors and lentiviruses. The specific performance depends on their characteristics and the target tissue. Non-viral based methods are usually less toxic and safer than viral vectors, but gene expression may be less efficient. Common methods include nanoparticles, liposomes, etc. Our development focus is to combine the safety of non-viral approaches with the efficiency of viral vectors to achieve ideal gene delivery for effective treatment of glaucoma.
Solutions for Viral Vectors
We offer adenovirus (AdV), adeno-associated virus (AAV) and retroviral vectors (RV) that are commonly used in gene therapy. AAV vectors are usually chosen, but we select the correct vector type with high transduction efficiency depending on the type of target tissue. A representative animal model will be selected to analyze the effect.
Solutions for Non-viral Vectors
In order to protect therapeutic nucleic acids for safe and effective delivery, we offer services for the development of non-viral vector delivery methods. These different chemicals are used to deliver across the cell membrane, and specific protocols can be found below.
- Nanoparticle systems
Nanodelivery systems are powerful non-viral delivery vehicles. We offer nanotechnology applications, and the possibility to modify our services extensively to improve delivery function and tissue targeting. - Lipid-based systems
Liposomes are the most common lipid-based gene delivery system. We can design liposomal systems with details such as molecular composition, structure, charge ratio, and head group to form lipid complexes with high transfection efficiency. Of course, we can also modify groups and combine ligands according to customer's specific requirements to improve transfection efficiency and reduce cytotoxicity. - Polymer-based systems
Polymers are long chain structures composed of repeating monomeric small molecules. Different types of synthetic and natural polymers can be evaluated as gene delivery carriers based on their biocompatibility and safety. Polymers such as chitosan, PEI, PLGA, etc. have been used for gene drug delivery. We are able to employ three commonly used strategies to produce polymer-based vectors and characterize the chemical, biological and mechanical properties of the polymers.
The success of gene therapy for glaucoma relies on effective and stable gene delivery systems. We offer a variety of currently viable gene therapy delivery strategies, focusing on our clients' delivery challenges in the development of gene therapies for glaucoma.
If you would like to learn more about our gene delivery specific services, please contact us.
References
- Kaufman, et al. Glaucoma Drugs in the Pipeline. Asia-Pacific Journal of Ophthalmology, 2018, 7(5):345-351.
- Ana V, et al. Non-viral strategies for ocular gene delivery. Materials Science and Engineering C, 2017.